Amryt claims a win in epidermolysis bullosa

Hopes that Amryt’s Filsuvez (AP101) might show efficacy in epidermolysis bullosa had dimmed after the readout from the pivotal Ease trial was delayed, in part to increase enrolment. So today’s announcement of a hit on the primary endpoint came as a surprise, sending shares in the London and Nasdaq-listed company up 40%, to a record high. Significantly more patients in the Filsuvez arm had complete closure of the target wound within 45 days (p=0.013) than those on placebo, Amryt said. Secondary measures failed to hit statistical significance, although “favourable differences” were observed. Puzzlingly, considering the hit on the primary, the key secondary of time to target wound closure over 90 days was not met. Still, executives claimed on a call that Filsuvez “undoubtedly had an effect on acceleration of wound healing”. Full presentation of the data will be keenly awaited to see how close these missed endpoints came, while the numbers behind the primary endpoint also remain important. Still, given the intractable nature of this devastating skin disease, and lack of options, Filsuvez seems to have a good chance of getting to market, something Amryt hopes to see happen before the end of 2021.

The Ease trial: awaiting further details. 
Primary endpoint Outcome
Proportion of patients with complete closure of the target wound within 45 ± 7 days of treatment Hit (p=0.013)
Key secondary endpoint  
Time to wound closure up to 90 ± 7 days of treatment Missed
Other secondary endpoints  
Incidence of first complete wound closure of EB target wound at different time points Missed
Change from baseline in EB target wound size Missed
Change in total body wound burden over time Missed
Change in percentages of TBSA affected by EB partial thickness wounds Missed
Patient reported outcomes   
Change from baseline in background and procedural pain after wound dressing change  
Change from baseline in itching before wound dressing change  
Response to treatment  
Change from baseline in sleep quality  
Number of days missed from school or work  
Safety endpoints  
Incidence, severity, and relatedness of AEs  
Local tolerability  
Laboratory findings  
Incidence and severity of wound infections  
Source: Study design and rationale.

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